AT-007 in Adult Subjects With Classic Galactosemia (CG)

• Status: Active, not recruiting
• Trial end date: 2022-12-01
• Target enrollment: 0
• Participant gender: All

Summary

This study is a 12-month open-label extension (OLE) study of AT-007 in adult subjects with CG who previously participated in Study AT-007-1001 Part D and/or Part D Extension.

The study is designed to assess the long-term safety of AT-007 in subjects with CG as well as the pharmacodynamics (PD) (inhibition of galactitol) and PK of AT-007. The effect of 12-month treatment with AT-007 on the levels of galactose and other galactose metabolites in subjects with CG will also be evaluated.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Applied Therapeutics, Inc.

Criteria

Inclusion Criteria:

• Male or non-pregnant, non-lactating female subject with a CG diagnosis

• Previous participation in Study AT-007-1001 Part D and/or Part D Extension

• No other significant health problems which preclude participation

Exclusion Criteria:

• Concomitant use of certain medications or over-the-counter therapies

• Discontinuation from Study AT-007-1001 Part D and/or Part D Extension due an adverse event